Galimedix Therapeutics announced the successful completion of its Phase 1 clinical trial for GAL-101, an oral small molecule designed to target misfolded amyloid beta (Aβ) monomers in Alzheimer's disease. The study, which enrolled over 100 healthy volunteers, demonstrated that GAL-101 was well tolerated with no serious adverse events (SAEs) observed and showed a highly favorable safety profile.
The Phase 1 trial evaluated the safety, tolerability, and pharmacokinetics of single ascending dose (SAD) and multiple ascending dose (MAD) oral administrations of GAL-101. Crucially, the study confirmed that GAL-101 effectively crosses the blood-brain barrier, consistent with pre-clinical findings, and its pharmacokinetic profile strongly supports advancing the oral formulation into Phase 2 development.
Novel Mechanism of Action
GAL-101 represents a unique approach to Alzheimer's treatment by specifically targeting misfolded Aβ monomers to prevent the formation of toxic Aβ oligomers and protofibrils. This mechanism differs from current approved therapies and addresses what many studies have indicated are major underlying causes of neurodegenerative diseases affecting both the brain and retina.
In pre-clinical testing, GAL-101 demonstrated the ability to prevent and eliminate all forms of toxic Aβ species while leaving healthy Aβ forms intact. The compound also showed potential for neuroprotection and symptomatic alleviation in pre-clinical models of Alzheimer's disease, with no antibody-specific immunological side effects such as ARIA (amyloid-related imaging abnormalities).
Clinical Development Strategy
"Completing our first clinical trial with the oral formulation of GAL-101 is an important milestone for Galimedix," said Alexander Gebauer, MD, PhD, Co-founder and Executive Chairman of Galimedix. "We are pleased that the results showed that oral GAL-101 was well tolerated with a highly favorable safety profile."
The company is now planning a Phase 2 trial in Alzheimer's disease, targeting a patient population with very limited treatment options. Gebauer noted that "GAL-101 is expected to be first-in-class and has the potential to become the future standard of care for all stages of Alzheimer's, including mild cognitive impairment."
The Phase 1 study also evaluated various parameters including the effects of food, age, and gender - all relevant aspects required to initiate the planned Phase 2 study in Alzheimer's disease. Fundraising has been initiated to support the next phase of development.
Parallel Development in Ophthalmology
Simultaneously, Galimedix is conducting a Phase 2 clinical trial with GAL-101 eyedrops for dry age-related macular degeneration (dAMD) in the eDREAM study (NCT06659549). Recruitment is ongoing in the US, Europe, and other regions, with this development being partner-funded, which mitigates Galimedix's financial exposure.
In pre-clinical ophthalmic models, GAL-101 demonstrated strong efficacy in protecting neuronal retinal cells from toxic damage. A previous Phase 1 study with GAL-101 eyedrops showed an excellent safety and tolerability profile.
Manufacturing and Commercial Advantages
GAL-101 offers several potential advantages including robust storage stability and easy, inexpensive manufacturing. The compound has shown very low systemic toxicity in pre-clinical studies, supporting its development as both an oral and topical formulation.
The company plans to present full study results at a future scientific conference and is actively seeking discussions with interested parties regarding investment and partnering opportunities at upcoming industry conferences including the LSX World Congress USA, Biotech in Europe Forum, BIO-Europe, and LSX Invεstival.
